{"id":5457,"date":"2026-01-12T16:46:39","date_gmt":"2026-01-12T13:46:39","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5457"},"modified":"2026-01-12T16:46:40","modified_gmt":"2026-01-12T13:46:40","slug":"precision-biosciences-anticipates-beginning-patient-dosing-for-duchenne-muscular-dystrophy-in-the-early-second-quarter-of-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-anticipates-beginning-patient-dosing-for-duchenne-muscular-dystrophy-in-the-early-second-quarter-of-2026\/","title":{"rendered":"Precision BioSciences, Duchenne Musk\u00fcler Distrofisi Hastalar\u0131na Doz Uygulamas\u0131na 2026 Y\u0131l\u0131n\u0131n \u0130kinci \u00c7eyre\u011finin Ba\u015flar\u0131nda Ba\u015flamay\u0131 Bekliyor"},"content":{"rendered":"

DMD program\u0131 PBGENE-DMD i\u00e7in Precision BioSciences, beklenen IND onay\u0131n\u0131n ard\u0131ndan 2026 y\u0131l\u0131n\u0131n ilk \u00e7eyre\u011finin sonlar\u0131nda veya ikinci \u00e7eyre\u011finin ba\u015flar\u0131nda hasta dozlamas\u0131na ba\u015flamay\u0131 \u00f6ng\u00f6r\u00fcyor.<\/strong> Faz 1\/2 FUNCTION-DMD \u00e7al\u0131\u015fmas\u0131, 45-55. ekzonlarda mutasyon bulunan ayakta tedavi g\u00f6ren hastalar\u0131 tedavi edecektir. Birden fazla hastadan elde edilen ilk verilerin 2026 y\u0131l\u0131n\u0131n sonuna kadar elde edilmesi beklenmektedir.<\/p>\n\n\n\n

PBGENE-DMD \u2013 Duchenne Kas Distrofisi (DMD) Hastalar\u0131nda Faz 1\/2 FUNCTION-DMD \u00c7al\u0131\u015fmas\u0131<\/h2>\n\n\n\n

PBGENE-DMD, s\u0131n\u0131f\u0131nda ilk olan yeni bir gen d\u00fczenleme terapisi olup, mevcut mikrodistrofin ve ekzon atlama tedavilerinden a\u00e7\u0131k\u00e7a farkl\u0131la\u015fan bir gen kesme yakla\u015f\u0131m\u0131 kullanmaktad\u0131r. PBGENE-DMD, DMD hastalar\u0131nda 45-55. ekzonlarda mutasyon bulunan ve DMD'li erkek \u00e7ocuklarda 60%'ye kadar g\u00f6r\u00fclen hastalarda kal\u0131c\u0131 fonksiyonel kas iyile\u015fmesi sa\u011flama potansiyeline sahip olacak \u015fekilde tasarlanm\u0131\u015ft\u0131r.<\/strong> Tek bir AAV, hastan\u0131n DNA's\u0131ndaki distrofin genini kal\u0131c\u0131 olarak d\u00fczenlemek \u00fczere tasarlanm\u0131\u015f iki ARCUS proteinini kodlar ve bu da do\u011fal olarak ifade edilen, neredeyse tam uzunlukta, i\u015flevsel bir distrofin proteiniyle sonu\u00e7lan\u0131r. G\u00fc\u00e7l\u00fc preklinik kan\u0131tlarla desteklenen PBGENE-DMD, kas uydu h\u00fccrelerini hedefleyerek zaman i\u00e7inde i\u015flevsel iyile\u015fmeyi sa\u011flamak \u00fczere tasarlanm\u0131\u015ft\u0131r. Daha fazla bilgi edin:<\/strong> Precision BioSciences PBGENE-DMD, Neredeyse Tam Uzunlukta Distrofin Proteininin \u00dcretimini Geri Kazand\u0131r\u0131r<\/a><\/p>\n\n\n\n

Ara\u015ft\u0131rma ama\u00e7l\u0131 yeni ila\u00e7 (IND) ba\u015fvurusunun onaylanmas\u0131n\u0131n ard\u0131ndan, FUNCTION-DMD Faz 1\/2 klinik denemesine ilk hastan\u0131n 2026 y\u0131l\u0131n\u0131n ilk \u00e7eyre\u011finin sonlar\u0131nda veya ikinci \u00e7eyre\u011finin ba\u015flar\u0131nda dahil edilmesi bekleniyor. Bu \u00e7al\u0131\u015fma, d\u00fcnya standartlar\u0131nda uzmanla\u015fm\u0131\u015f DMD klinik merkezlerinde, 45-55. ekzonlarda mutasyon bulunan ayakta tedavi g\u00f6ren hastalar\u0131 tedavi etmek i\u00e7in uygun bir imm\u00fcn mod\u00fclasyon rejimi ve g\u00fcvenlik izleme program\u0131 kullanacakt\u0131r. \u00c7ok say\u0131da hastadan elde edilen ilk verilerin 2026 y\u0131l sonuna kadar elde edilmesi beklenmekte olup, erken etkinlik, kas biyopsilerinden elde edilen neredeyse tam uzunluktaki distrofin protein ekspresyonunun y\u00fczdesiyle de\u011ferlendirilecektir.<\/strong> En az 10 DMD hastas\u0131ndan elde edilen destekleyici verilerin ard\u0131ndan \u015firket, d\u00fczenleyici s\u00fcre\u00e7le ilgili ileriye d\u00f6n\u00fck bir yol haritas\u0131 belirlemek \u00fczere FDA ile bir araya gelecekti.<\/p>\n\n\n\n

Devam\u0131n\u0131 oku<\/strong>: Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"

For the DMD program PBGENE-DMD, Precision BioSciences anticipates starting patient dosing in late first or early second quarter of 2026, following expected IND approval. The Phase 1\/2 FUNCTION-DMD study will treat ambulatory patients with mutations in exons 45-55. Initial data from multiple patients are expected by the end of 2026. PBGENE-DMD – Phase 1\/2 FUNCTION-DMD […]<\/p>\n","protected":false},"author":1,"featured_media":5458,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[72,314,313],"class_list":["post-5457","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5457","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5457"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5457\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5458"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5457"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5457"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5457"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}